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Open AccessJournal ArticleDOI

Phase II Trial of the CDK4 Inhibitor PD0332991 in Patients With Advanced CDK4-Amplified Well-Differentiated or Dedifferentiated Liposarcoma

TLDR
Treatment with theCDK4 inhibitor PD0332991 was associated with a favorable progression-free rate in patients with CDK4-amplified and RB-expressing WDLS/DDLS who had progressive disease despite systemic therapy.
Abstract
Purpose CDK4 is amplified in 90% of well-differentiated (WDLS) and dedifferentiated liposarcomas (DDLS). The selective cyclin-dependent kinase 4 (CDK4)/CDK6 inhibitor PD0332991 inhibits growth and induces senescence in cell lines and xenografts. In a phase I trial of PD0332991, several patients with WDLS or DDLS experienced prolonged stable disease. We performed an open-label phase II study to determine the safety and efficacy of PD0332991 in patients with advanced WDLS/DDLS.

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Targeted treatments of sarcomas and connective tumors beside gastrointestinal stromal tumor.

TL;DR: Several targeted therapies are in development in sarcoma and could be added to the therapeutic armamentarium in the near future, however, predictive factors still need to be identified to better select the target population of these new drugs.
Patent

HSPC-Sparing Treatments for RB-Positive Abnormal Cellular Proliferation

TL;DR: In this article, improved compounds for and methods of treating selected RB-positive cancers and other Rb-positive abnormal cellular proliferative disorders while minimizing the deleterious effects on healthy cells, for example healthy Hematopoietic Stem Cells and Progenitor Cells (HSPCs), associated with current treatment modalities.
Journal ArticleDOI

Comprehensive Genomic Analysis Reveals the Prognostic Role of LRRK2 Copy-Number Variations in Human Malignancies

TL;DR: The results suggest that both L RRK2 amplifications and whole gene deletions could play a role in cancer development, paving the way for future research in terms of potential treatment with LRRK2 small molecule inhibitors for LRRk2-amplified cases.
Journal ArticleDOI

Advances in the Pathology and Molecular Biology of Sarcomas and the Impact on Treatment.

TL;DR: An overview of the pathology, coupled with recent advances in molecular biology, of a selection of soft tissue sarcomas from a clinicopathological perspective is given, discussing histopathological diagnosis with developments in molecular diagnosis and the incorporation of these findings into diagnostic practice and current and potential targeted treatments.
References
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Book

Statistical methods for rates and proportions

TL;DR: In this paper, the basic theory of Maximum Likelihood Estimation (MLE) is used to detect a difference between two different proportions of a given proportion in a single proportion.
Journal ArticleDOI

Statistical Methods for Rates and Proportions.

B. S. Everitt, +1 more
- 01 Sep 1973 - 
Journal Article

[New response evaluation criteria in solid tumours-revised RECIST guideline (version 1.1)].

TL;DR: This paper is an overview of the new response evaluation criteria in solid tumours: revised RECIST guideline (version 1. 1), with a focus on updated contents.
Journal Article

Specific inhibition of cyclin-dependent kinase 4/6 by PD 0332991 and associated antitumor activity in human tumor xenografts

TL;DR: Results indicate that inhibition of Cdk4/6 alone is sufficient to cause tumor regression and a net reduction in tumor burden in some tumors.
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